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This is an investigational allogeneic hematopoietic stem cell transplantation (HSCT) protocol developed by the University of Illinois at Chicago (UIC) specifically for the treatment of adults with high-risk or aggressive sickle cell disease (SCD). The therapy involves the infusion of HLA-matched related donor hematopoietic stem cells following a non-myeloablative conditioning regimen. This regimen consists of the monoclonal antibody alemtuzumab (targeting CD52) and low-dose total body irradiation (300 cGy), designed to achieve stable mixed chimerism—a state where donor and recipient cells coexist—without the severe toxicity associated with traditional high-dose myeloablative chemotherapy. Post-transplant, patients receive sirolimus for immunosuppression. The primary goal is to replace the recipient's defective erythropoiesis with healthy donor cells to reverse the sickle cell phenotype while minimizing transplant-related morbidity and mortality.
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